On May 19, 2021 NuCana plc (NASDAQ: NCNA) reported that financial results for the first quarter ended March 31, 2021 and provided an update on its broad clinical program with its transformative ProTide therapeutics (Press release, Nucana BioPharmaceuticals, MAY 19, 2021, View Source [SID1234584207]).

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As of March 31, 2021, NuCana had cash and cash equivalents of £78.6 million compared to £87.4 million as of December 31, 2020. NuCana continues to advance its various clinical programs and reported a net loss of £9.8 million for the quarter ended March 31, 2021, as compared to a loss of £4.0 million for the quarter ended March 31, 2020. Basic and diluted loss per share was £0.19 for the quarter ended March 31, 2021, as compared to £0.12 per share for quarter ended March 31, 2020.

"We are very pleased with our momentum in 2021," said Hugh S. Griffith, NuCana’s Founder and Chief Executive Officer. "In January, we presented data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO-GI) from the NuTide:302 study demonstrating NUC-3373’s encouraging efficacy signals and favorable safety profile in patients with advanced colorectal cancer. Among the efficacy-evaluable population, a disease control rate of 62% was achieved. In addition, NUC-3373 was well tolerated with no hand-foot syndrome or neutropenia as well as lower rates of diarrhea, mucositis and stomatitis as compared to historical data for 5-FU and capecitabine in the frontline treatment of patients with colorectal cancer."

Mr. Griffith continued: "In April, we were excited to present five posters at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. NUC-3373 maintained the encouraging 62% disease control rate in the efficacy-evaluable population in the NuTide:302 study. The poster also detailed three patients who experienced reductions in their target lesions of 40%, 28% and 15% and several patients who achieved a longer progression-free survival on NUC-3373 than they had on their prior therapy. We also presented additional clinical data from the ongoing Phase I study of NUC-7738. These data demonstrated NUC-7738’s encouraging anti-cancer activity and favorable tolerability profile. Three case studies described patients who achieved tumor volume reductions and prolonged stable disease on NUC-7738. Other AACR (Free AACR Whitepaper) posters showed NUC-3373-treated colon cancer cells are able to activate a natural killer cell response and described how NUC-7738 was designed to overcome the key cancer resistance mechanisms which have prevented the clinical development of its parent nucleoside analog, 3’-deoxyadenosine. Overall, these presentations highlighted the potential of our ProTides to significantly improve the treatment outcomes for patients with cancer."

Mr. Griffith concluded: "We are excited with the progress we have made so far in 2021. We remain focused on continuing to drive recruitment across all of our ongoing studies, including our Phase III study of Acelarin plus cisplatin in patients with biliary tract cancer as well as initiating new studies, including our second Phase III study evaluating NUC-3373 in combination with other agents for patients with colorectal cancer. We look forward to providing additional updates as we go through 2021."

Anticipated 2021 Milestones

Acelarin is a ProTide transformation of gemcitabine. In 2021, NuCana expects to:
Complete recruitment sufficient to enable the first interim analysis in 2022 of the Phase III study of Acelarin combined with cisplatin as a first-line treatment for patients with advanced biliary tract cancer.

NUC-3373 is a ProTide transformation of the active anti-cancer metabolite of 5-FU. In 2021, NuCana expects to:
Report data from the Phase Ib study (NuTide:302) of NUC-3373 in combination with other agents with which 5-FU is typically combined, such as leucovorin, oxaliplatin and irinotecan in patients with advanced colorectal cancer;
Initiate and report data from a Phase Ib expansion / Phase II study of NUC-3373 in combination with other agents for patients with colorectal cancer;
Initiate a Phase III study of NUC-3373 in combination with other agents for patients with colorectal cancer; and
Report data from the Phase I study (NuTide:301) of NUC-3373 in patients with advanced solid tumors.

NUC-7738 is a ProTide transformation of a novel nucleoside analog, 3’-deoxyadenosine. In 2021, NuCana expects to:
Report data from the Phase I study (NuTide:701) of NUC-7738 in patients with advanced solid tumors; and
Initiate a Phase II study of NUC-7738 in patients with solid tumors.

On May 19, 2021 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics, reported that the Company will present a Trials in Progress poster presentation for its lead artificial antigen-presenting (aAPC) cell program, RTX-321, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held virtually from June 4-8, 2021 (Press release, Rubius Therapeutics, MAY 19, 2021, View Source [SID1234584706]).

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The Trials in Progress poster presentation will summarize the proposed mechanism of action of RTX-321, preclinical observations to date and the clinical trial design, including translational medicine methodology, for the Company’s ongoing Phase 1 clinical trial of RTX-321 for the treatment of HPV 16-positive cancers, including cervical cancer, anal cancer, and head and neck cancer.

Poster Title: A Phase 1 Study of RTX-321, an Engineered Red Blood Cell as an Artificial Antigen-Presenting Cell Expressing HLA-A*02 with the HPV-16 E7 Peptide and 4-1BB Ligand with Membrane-Bound IL-12 for the Treatment of HPV 16-Positive Cancers
Session Title: Poster Session: Developmental Therapeutics—Immunotherapy
Abstract Number: TPS2664
Date and Time: Friday, June 4, 2021 at 9:00 AM ET on the ASCO (Free ASCO Whitepaper) website, View Source

About HPV 16-Positive Cancers
Human papillomavirus (HPV) 16 is associated with approximately 70 percent of cervical cancers, approximately 40 percent of head and neck squamous cell carcinoma (HNSCC) arising in the oropharynx, approximately 25-40 percent of HNSCC arising in other locations and approximately 80-85 percent of anal cancers. A critical need remains for better treatment options for advanced HPV 16-associated cancers. The prognosis remains poor for patients with metastatic disease with few treatment options beyond the first-line setting.

About the RTX-321 Clinical Trial
Rubius Therapeutics is enrolling patients in a Phase 1 open-label, multicenter, monotherapy dose escalation, first-in-human study of RTX-321 for the treatment of patients that are HLA-A*02:01-positive with persistent, recurrent, or metastatic, unresectable, HPV 16-positive cancers, including unresectable cervical cancer (squamous, adeno, or adenosquamous histology), head and neck squamous cell carcinoma (including of the nasal and oral cavities, larynx, hypopharynx, nasopharynx, and oropharynx) and squamous cell cancer of the anal canal that is not amenable to curative therapy. The purpose of the trial is to determine the safety and tolerability, recommended Phase 2 dose and pharmacology, and antitumor activity of RTX-321. For more information about the Phase 1 clinical trial of RTX-321, please visit clinicaltrials.gov (NCT04672980).

About RTX-321
RTX-321 is an allogeneic, off-the-shelf aAPC therapy product candidate that is engineered to induce a tumor-specific immune response by expanding antigen-specific T cells. RTX-321 expresses hundreds of thousands of copies of an HPV peptide antigen bound to major histocompatibility complex class I proteins, the costimulatory molecule 4-1BBL and the cytokine IL-12 on the cell surface to mimic human T cell-APC interactions.

On May 19, 2021 Genmab A/S (Nasdaq: GMAB) reported that In accordance with Article 19 of Regulation No. 596/2014 on Market Abuse and Implementing Regulation 2016/523, this document discloses the data of the transactions made in Genmab A/S (Nasdaq: GMAB) made by managerial employees and their closely associated persons (Press release, Genmab, MAY 19, 2021, View Source [SID1234580239]).

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The company’s managerial employees and their closely associated persons have given Genmab A/S power of attorney on their behalf to publish trading in Genmab shares by the company’s managerial employees and their closely associated persons.

On May 19, 2021 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies, reported that additional data from its Phase 1/2a study of PBCAR0191, the Company’s first generation, off-the-shelf allogeneic CAR T candidate targeting CD19 will be presented at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Meeting being held virtually June 4-8, 2021 (Press release, Precision Biosciences, MAY 19, 2021, View Source [SID1234580255]).

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Abstracts are available and can be viewed on the ASCO (Free ASCO Whitepaper) web site at www.asco.org. Additional data including updated response rates, safety, and durability will be presented at the ASCO (Free ASCO Whitepaper) meeting.

Title: Preliminary safety and efficacy of PBCAR0191, an allogeneic, off-the-shelf CD19-targeting CAR T product, in relapsed/refractory (r/r) CD19+ NHL
Poster Session: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia, Abstract 7516
Date/Time: Friday, June 4, 2021 at 9:00 a.m. ET
Presenting Author: Bijal Shah, M.D., Moffitt Cancer Center

By February 1, 2021, 13 patients with R/R non-Hodgkin lymphoma (NHL) met study eligibility criteria and had received PBCAR0191 dose level 3 at 3×106 or equivalent with either standard lymphodepletion (sLD) (n=6)1 or enhanced lymphodepletion (eLD) (n=7)2. Of these patients, 77% had aggressive lymphomas and 62% had four or more courses of prior treatment. PBCAR0191 continued to demonstrate an acceptable safety profile with no cases of graft versus host disease, no cases of Grade ≥ 3 cytokine release syndrome, and no cases of Grade ≥ 3 immune effector cell-associated neurotoxicity syndrome.

"These results continue to confirm the activity of PBCAR0191 with strategies that mitigate rejection, and we look forward to sharing additional data in the poster and on our hosted conference call on June 4," said Alan List, M.D., Chief Medical Officer at Precision BioSciences. "While we continue to advance PBCAR0191 through its Phase 1/2a study, we eagerly await the clinical start of our next-generation, immune evading stealth cell technology with PBCAR19B, which includes modifications designed to enhance CAR T persistence and delay allogeneic immune rejection by T cells and natural killer cells."

Company-Hosted Conference Call and Web Cast Information
Precision will host a conference call and webcast on Friday, June 4, 2021 at 8:00 a.m. ET to review the most recent interim clinical data for PBCAR0191. The dial-in conference call numbers for domestic and international callers are (866) 996-7202 and (270) 215-9609, respectively. The conference ID number for the call is 5647916. Participants may access the live webcast and the accompanying presentation materials on Precision’s website www.precisionbiosciences.com in the Investors and Media section under Events and Presentations. An archived replay of the webcast will be available on Precision’s website.

On May 19, 2021 Advaxis, Inc. (Nasdaq: ADXS), a clinical-stage biotechnology company focused on the development and commercialization of immunotherapy products reported updated data from the Company’s ongoing Phase 1/2 study evaluating ADXS-503 in combination with KEYTRUDA which will be presented as a poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Annual Meeting (Press release, Advaxis, MAY 19, 2021, View Source [SID1234580272]). ADXS-503 is the first drug construct from the ADXS-HOT off-the-shelf, cancer-type specific, immunotherapy program which leverages Advaxis’ proprietary Lm technology platform to target hotspot mutations that commonly occur in specific cancer types as well as other proprietary, tumor-associated antigens.

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"These early results, which include a disease control rate of 44% in the first 9 evaluable patients treated with ADXS-503 as an add on therapy at progression with pembrolizumab, are particularly encouraging given the durable nature of disease control in 3 patients," said Suresh Ramalingam, M.D., Roberto C. Goizueta Chair for Cancer Research, Director, Division of Medical Oncology and Deputy Director of the Winship Cancer Institute at Emory University School of Medicine. "The translational results from the study provide interesting insights on the potential mechanism of action by which the addition of ADXS-503 may may be effective in this patient population."

Ken Berlin, Chief Executive Officer of Advaxis, said, "We are encouraged by these updated results which continue to support the potential of ADXS-503 to enhance and/or restore sensitivity to checkpoint inhibitors. The clinical activity observed to date in this challenging patient population, combined with a favorable safety profile, suggest that ADXS-503 may be an important new off-the-shelf immunotherapy treatment option. We look forward to continued progress in the clinic with our ADXS-HOT products which includes the ongoing Phase 1/2 study of ADXS-503 in NSCLC, and the expansion of the program to our planned Phase 1 Study of ADXS-504 for the treatment of early prostate cancer."

Key presentation highlights:

Highlights from the poster presentation titled, "A phase 1 study of an off-the-shelf, multi-neoantigen vector (ADXS-503) in patients with metastatic non-small-cell lung cancer (NSCLC) progressing on pembrolizumab as last therapy" presented by Missak Haigentz, M.D., Section Chief of Hematology and Oncology at Morristown Medical Center and Medical Director of Hematology and Oncology for Atlantic Health, and Study Investigator, include:

10 patients have been treated with ADXS-503 as an add on therapy to patients failing pembrolizumab as last therapy with 10 patients evaluable for safety and 9 patients evaluable for efficacy

Combination therapy was well tolerated with no DLTS or added toxicity of the two drugs. Grade 1 and 2, transient and reversible events included chills, fever, fatigue, in approximately half of the patients

The Disease Control Rate (DCR) was 44% (4/9)

Clinical benefit was durable, with an observed partial response (PR) and stable disease (SD) sustained for over a year, and another observed SD lasting over 6 months. An additional PR was maintained for approximately 4 months

Biomarker data demonstrate that patients who seem to achieve clinical benefit include those with PD-L1 expression ≥50%, secondary resistance disease to pembrolizumab and those who show proliferation and/or activation of NK and CD8+ T cells within the first weeks of therapy

Translational studies show:

Antitumoral T-cell responses elicited against hot-spot mutation antigens and/or tumor associated antigens (TAAs)

Emergence of naive CD8+ Tcell clones, suggesting reactivity against novel antigens

Induction of proliferation and/or activation of pre-existing CD8+ T-cell clones, including PD-1 upregulation

Enrollment in Part B of the ongoing study will continue to further evaluate the clinical benefit and immune effects of adding on ADXS-503 to patients progressing on pembrolizumab
The Phase 1/2 clinical trial of ADXS-503 is seeking to establish the recommended dose, safety, tolerability and clinical activity of ADXS-503 administered alone and in combination with a KEYTRUDA in approximately 50 patients with NSCLC, in at least five sites across the U.S. The two dose levels with monotherapy in Part A, (1 X108 and 5 X108 CFU) have been completed. Part B with ADXS-503 (1 X108 CFU) in combination with KEYTRUDA is currently enrolling its efficacy expansion for up to 18 patients at dose level 1 (1 X108 CFU + KEYTRUDA) with the potential to proceed to dose level 2 (5 X108 CFU + KEYTRUDA) at a later date. Part C, which is evaluating ADXS-503 in combination with KEYTRUDA (1 X108 CFU + KEYTRUDA) as a first line treatment for patients with NSCLC with PD-L1 expression ≥ 1% or who are unfit for chemotherapy is currently enrolling patients.

About ADXS-HOT
ADXS-HOT is a program that leverages the Company’s proprietary Lm technology to target hotspot mutations that commonly occur in specific cancer types. ADXS-HOT drug candidates are designed to target acquired shared or "public" mutations in tumor driver genes along with other proprietary cancer-testes and oncofetal tumor-associated antigens that also commonly occur in specific cancer types. ADXS-HOT drug candidates are an off-the-shelf treatment, designed to potentially treat all patients with a specific cancer type, without the need for pretreatment biomarker testing, DNA sequencing or diagnostic testing.